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Bluebird Bio, now rebranded as Genetix Biotherapeutics, is a commercial-stage biotechnology company.
They appear twice among the makers of the 10 most expensive drugs in the U.S.
Skysona, which treats inherited brain disease (cerebral adrenoleukodystrophy), costs $3 million, while Zynteglo, a treatment for inherited blood disorder (beta-thalassemia), costs $2.8 million.
The FDA approved both therapies on September 16, 2022. Each is a one-time gene therapy made using the patient’s own stem cells.
They remove the cells, genetically modify them outside the body, and then infuse them back into the patient.
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